#CLN1 disease is caused by a deficiency of palmitoyl-protein thioesterase 1 (PPT1). This is one of the most aggressive childhood neurodegenerative diseases with unmet medical needs. Here, the authors provide evidence of the efficacy of a novel approach of #HematopoieticStemCell -based #GeneTherapy in the #CLN1 mouse model.

By M. Peviani, A. Biffi & colleagues

#Openaccess: https://doi.org/10.15252/emmm.202215968